Primary Purpose
| primary_purpose | Study_Count |
|---|---|
| Treatment | 55 |
| Diagnostic | 7 |
| Basic Science | 2 |
| NA | 2 |
| Other | 1 |
| Supportive Care | 1 |
Craig Mayer, Nick WIlliams, Vojtech Huser
Lister Hill National Center for Biomedical Communications, National Library of Medicine, NIH, Bethesda, MD
See the full regCTG repository at https://github.com/lhncbc/CRI/tree/master/regCTG
To return to the list of conditions and view other reports go to https://lhncbc.github.io/CRI/regCTG/regCTG-condition_report_links.html
| Type | Study_count |
|---|---|
| Total_Studies | 116 |
| interventional | 68 |
| Observational | 43 |
| Registry | 5 |
```
| intervention_type | Study_Count |
|---|---|
| Drug | 31 |
| Biological | 18 |
| Genetic | 4 |
| Drug|placebo | 3 |
| Other | 3 |
| Biological|Drug | 2 |
| Device|Procedure | 2 |
| Biological|placebo | 1 |
| Device | 1 |
| Drug|Other|placebo | 1 |
| Other|Procedure | 1 |
| Procedure | 1 |
| Country | Study_Count |
|---|---|
| United States | 16 |
| Canada | 3 |
| France | 2 |
| Germany | 2 |
| NA | 2 |
| United States|Australia|Austria|Belgium|Canada|Germany|Ireland|Italy|Netherlands|Norway|Poland|Spain|Switzerland|United Kingdom | 2 |
| United States|Canada|Czech Republic|Hungary|Poland|United Kingdom | 2 |
| United States|France|Poland|Russian Federation|United Kingdom | 2 |
| Australia | 1 |
| Australia|Brazil | 1 |
| Australia|Canada|Czechia|Germany|Netherlands|Norway|Slovenia|Spain|United Kingdom | 1 |
| Australia|United Kingdom | 1 |
| Czech Republic|Estonia|Poland|Slovakia | 1 |
| Czechia | 1 |
| Denmark | 1 |
| France|United Kingdom | 1 |
| Italy | 1 |
| Italy|Norway|United Kingdom | 1 |
| Japan | 1 |
| Korea, Republic of | 1 |
| Netherlands | 1 |
| Spain | 1 |
| Switzerland | 1 |
| United Kingdom | 1 |
| United States|Argentina|Australia|Austria|Belgium|Brazil|Canada|Denmark|Egypt|France|Italy|Japan|Spain|Turkey|United Kingdom | 1 |
| United States|Argentina|Australia|Belgium|Brazil|Canada|Czechia|Finland|France|Germany|Hungary|Italy|Netherlands|Norway|Paraguay|Slovenia|Spain|Switzerland|Turkey|United Kingdom | 1 |
| United States|Argentina|Australia|Brazil|Canada|Denmark|Egypt|France|Italy|Poland|Spain|Turkey|United Kingdom | 1 |
| United States|Argentina|Brazil|Canada|Czech Republic|Netherlands|Norway|Poland|United Kingdom | 1 |
| United States|Australia|Austria|Belgium|Brazil|Denmark|France|Italy|Japan|United Kingdom | 1 |
| United States|Australia|Belgium|Canada|Netherlands | 1 |
| United States|Australia|Brazil|Canada|France|United Kingdom | 1 |
| United States|Australia|Canada | 1 |
| United States|Australia|Canada|Czechia|Hungary|Netherlands|Norway|Slovenia|Spain|United Kingdom | 1 |
| United States|Australia|Czechia|Finland|Paraguay|Poland|Slovenia|United Kingdom | 1 |
| United States|Australia|Czechia|Finland|Poland|Slovenia|United Kingdom | 1 |
| United States|Australia|Paraguay|Serbia|Spain|United Kingdom | 1 |
| United States|Austria|United Kingdom | 1 |
| United States|Belgium|Canada|Czechia|Denmark|Italy|Netherlands|Norway|Spain|Taiwan|Turkey|United Kingdom | 1 |
| United States|Belgium|Czechia|Denmark|Italy|Norway|United Kingdom | 1 |
| United States|Canada | 1 |
| United States|France|Netherlands|Puerto Rico|United Kingdom | 1 |
| United States|France|Poland|United Kingdom | 1 |
| United States|Paraguay|Spain|United Kingdom | 1 |
| United States|Spain | 1 |
| United States|Spain|United Kingdom | 1 |
| Site_count | Study_Count |
|---|---|
| 1 | 22 |
| 2 | 4 |
| 3 | 3 |
| 4 | 2 |
| 5 | 5 |
| 6 | 3 |
| 7 | 5 |
| 8 | 2 |
| 9 | 1 |
| 10 | 1 |
| 11 | 1 |
| 12 | 4 |
| 14 | 3 |
| 20 | 2 |
| 21 | 1 |
| 24 | 1 |
| 25 | 2 |
| 26 | 1 |
| 28 | 1 |
| 29 | 1 |
| 51 | 1 |
| 58 | 2 |
| Phase | Study_Count |
|---|---|
| Phase 3 | 17 |
| Phase 2 | 16 |
| N/A | 12 |
| Phase 1 | 10 |
| Phase 4 | 8 |
| Phase 1/Phase 2 | 5 |
| Number_of_Arms | Count_of_Studies |
|---|---|
| 1 | 43 |
| 2 | 13 |
| 3 | 3 |
| 4 | 1 |
| 5 | 2 |
| 6 | 1 |
| NA | 5 |
| Measure | N.A | Phase.1 | Phase.1.Phase.2 | Phase.2 | Phase.3 | Phase.4 |
|---|---|---|---|---|---|---|
| Min. | 12.0000 | 3.0 | 12 | 3.000 | 20 | 6.00 |
| 1st Qu. | 20.0000 | 6.0 | 15 | 8.750 | 31 | 18.75 |
| Median | 51.0000 | 11.0 | 15 | 14.000 | 58 | 26.50 |
| Mean | 141.0833 | 13.9 | 18 | 13.625 | 64 | 35.25 |
| 3rd Qu. | 109.2500 | 18.5 | 18 | 17.750 | 78 | 46.00 |
| Max. | 1000.0000 | 32.0 | 30 | 25.000 | 171 | 82.00 |
| group_type | Group_Count |
|---|---|
| Experimental | 79 |
| Active Comparator | 8 |
| Other | 6 |
| NA | 5 |
| Placebo Comparator | 4 |
| No Intervention | 1 |
| intervention_model | Study_Count |
|---|---|
| Single Group Assignment | 44 |
| Parallel Assignment | 16 |
| NA | 4 |
| Crossover Assignment | 2 |
| Sequential Assignment | 2 |
| primary_purpose | Study_Count |
|---|---|
| Treatment | 55 |
| Diagnostic | 7 |
| Basic Science | 2 |
| NA | 2 |
| Other | 1 |
| Supportive Care | 1 |
| Country | Study_Count |
|---|---|
| United States | 13 |
| France | 4 |
| Germany | 4 |
| NA | 3 |
| Denmark | 2 |
| Korea, Republic of | 2 |
| Argentina|Belgium|Croatia|Czechia|Denmark|France|Germany|United Kingdom | 1 |
| Austria | 1 |
| Austria|Belgium|Croatia|Finland|France|Georgia|Germany|Ireland|Italy|Malta|Poland|Portugal|Spain|United Kingdom | 1 |
| Austria|Croatia|France|Georgia|Germany|Poland|Portugal | 1 |
| Austria|Germany | 1 |
| Belgium | 1 |
| Belgium|Czech Republic|Denmark|Finland|Germany|Sweden | 1 |
| Czechia | 1 |
| Germany|India|Sri Lanka | 1 |
| Germany|Poland | 1 |
| Italy | 1 |
| Japan | 1 |
| Switzerland | 1 |
| United Kingdom | 1 |
| United States|Slovenia | 1 |
| Site_count | Study_Count |
|---|---|
| 1 | 30 |
| 2 | 3 |
| 3 | 1 |
| 6 | 1 |
| 9 | 2 |
| 11 | 1 |
| 14 | 1 |
| 16 | 1 |
| 20 | 1 |
| 22 | 1 |
| 45 | 1 |
| Measure | Observational |
|---|---|
| Min | 4.0000 |
| 1st Qu | 32.5000 |
| Median | 100.0000 |
| Mean | 526.1628 |
| 3rd Qu | 520.0000 |
| Max | 5000.0000 |
| observational_model | Study_Count |
|---|---|
| Cohort | 26 |
| NA | 9 |
| Case-Only | 6 |
| Case Control | 1 |
| Other | 1 |
| time_perspective | Study_Count |
|---|---|
| Prospective | 27 |
| NA | 6 |
| Cross-Sectional | 4 |
| Retrospective | 4 |
| Other | 2 |
| Country | Study_Count |
|---|---|
| Canada | 2 |
| United States | 2 |
| United States|Argentina|Australia|Belgium|Brazil|Bulgaria|Canada|Chile|China|Colombia|Croatia|Czechia|Denmark|Estonia|Finland|France|Germany|Hong Kong|Hungary|India|Indonesia|Italy|Japan|Korea, Republic of|Kuwait|Lithuania|Malaysia|Netherlands|Norway|Peru|Philippines|Poland|Portugal|Romania|Russian Federation|Singapore|Slovakia|Spain|Sweden|Taiwan|Thailand|United Kingdom | 1 |
| Site_count | Study_Count |
|---|---|
| 1 | 3 |
| 5 | 1 |
| 219 | 1 |
| Measure | Registries |
|---|---|
| Min | 8.0 |
| 1st Qu | 600.0 |
| Median | 4000.0 |
| Mean | 3721.6 |
| 3rd Qu | 5000.0 |
| Max | 9000.0 |
| observational_model | Study_Count |
|---|---|
| Cohort | 2 |
| Other | 2 |
| Case-Control | 1 |
| time_perspective | Study_Count |
|---|---|
| Prospective | 3 |
| Other | 2 |
| target_duration | Study_Count |
|---|---|
| 1 Day | 1 |
| 10 Years | 1 |
| 2 Years | 1 |
| 25 Years | 1 |
| 52 Weeks | 1 |
#If less then 500 trials
| nct_id | brief_title | link | overall_status | source | primary_completion_date |
|---|---|---|---|---|---|
| NCT03305250 | Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT03305250 | Recruiting | University Hospital Birmingham NHS Foundation Trust | 2022-01-31 |
| NCT03180840 | Study of the Safety, Efficacy, & PK of Pegunigalsidase Alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients | https://ClinicalTrials.gov/show/NCT03180840 | Active, not recruiting | Protalix | 2020-07-31 |
| NCT04455230 | A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190 | https://ClinicalTrials.gov/show/NCT04455230 | Recruiting | Freeline Therapeutics | 2026-12-31 |
| NCT03018730 | Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) | https://ClinicalTrials.gov/show/NCT03018730 | Active, not recruiting | Protalix | 2020-01-31 |
| NCT02995993 | Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT02995993 | Completed | Greenovation Biotech GmbH | 2017-10-09 |
| NCT02985710 | Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan | https://ClinicalTrials.gov/show/NCT02985710 | Recruiting | Massachusetts General Hospital | 2019-02-28 |
| NCT02930655 | A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease | https://ClinicalTrials.gov/show/NCT02930655 | Completed | Idorsia Pharmaceuticals Ltd. | 2016-02-01 |
| NCT02800070 | Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT02800070 | Active, not recruiting | University Health Network, Toronto | 2024-02-29 |
| NCT02795676 | Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function | https://ClinicalTrials.gov/show/NCT02795676 | Active, not recruiting | Protalix | 2021-10-31 |
| NCT02489344 | Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT02489344 | Completed | Sanofi | 2018-11-20 |
| NCT02450604 | Prevalence of Fabry’s Disease in a Population of Patients With Chronic Pain | https://ClinicalTrials.gov/show/NCT02450604 | Recruiting | University Hospital, Bordeaux | 2020-05-05 |
| NCT02228460 | Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT02228460 | Completed | Sanofi | 2016-09-30 |
| NCT02194985 | Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT02194985 | Active, not recruiting | Amicus Therapeutics | 2020-12-31 |
| NCT02090608 | Paricalcitol in Fabry Disease | https://ClinicalTrials.gov/show/NCT02090608 | Completed | Federico II University | 2013-12-31 |
| NCT02082327 | A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers | https://ClinicalTrials.gov/show/NCT02082327 | Completed | Amicus Therapeutics | 2014-06-30 |
| NCT01304277 | This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes. | https://ClinicalTrials.gov/show/NCT01304277 | Completed | Shire | 2012-12-31 |
| NCT01298141 | A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT01298141 | Completed | Shire | 2017-09-25 |
| NCT01997489 | Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients. | https://ClinicalTrials.gov/show/NCT01997489 | Completed | Rigshospitalet, Denmark | 2013-09-30 |
| NCT01981720 | Extension Study of PRX-102 for up to 60 Months | https://ClinicalTrials.gov/show/NCT01981720 | Active, not recruiting | Protalix | 2021-03-31 |
| NCT01947634 | Sleepiness and Sleep-disordered Breathing in Fabry Disease. A Prospective Cohort Study. | https://ClinicalTrials.gov/show/NCT01947634 | Completed | University of Zurich | 2015-01-31 |
| NCT01853852 | A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects | https://ClinicalTrials.gov/show/NCT01853852 | Completed | Amicus Therapeutics | 2011-12-31 |
| NCT01730482 | A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014) | https://ClinicalTrials.gov/show/NCT01730482 | Completed | Amicus Therapeutics | 2011-09-30 |
| NCT01730469 | Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function | https://ClinicalTrials.gov/show/NCT01730469 | Completed | Amicus Therapeutics | 2012-04-30 |
| NCT01678898 | Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients | https://ClinicalTrials.gov/show/NCT01678898 | Completed | Protalix | 2016-03-06 |
| NCT01653444 | Evaluate the Safety and Exploratory Efficacy of GC1119 | https://ClinicalTrials.gov/show/NCT01653444 | Completed | Green Cross Corporation | 2014-05-31 |
| NCT01650779 | A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta | https://ClinicalTrials.gov/show/NCT01650779 | Completed | Sanofi | 2013-03-31 |
| NCT04002531 | A One Visit Follow Up of Adults With Fabry Disease Who Started Long-term Enzyme Replacement Therapy As Children | https://ClinicalTrials.gov/show/NCT04002531 | Enrolling by invitation | Baylor Research Institute | 2019-12-31 |
| NCT01489995 | Migalastat Food Effect Study | https://ClinicalTrials.gov/show/NCT01489995 | Completed | Amicus Therapeutics | 2011-12-31 |
| NCT01363492 | Safety Study of Replagal® Therapy in Children With Fabry Disease | https://ClinicalTrials.gov/show/NCT01363492 | Completed | Shire | 2013-05-31 |
| NCT04143958 | To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease | https://ClinicalTrials.gov/show/NCT04143958 | Recruiting | Sanofi | 2023-11-30 |
| NCT04073888 | Study of the Spermatic Characteristics of Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT04073888 | Completed | University Hospital, Bordeaux | 2013-10-18 |
| NCT04050137 | Therapeutic Exercise to Treat Neuropathic Pain | https://ClinicalTrials.gov/show/NCT04050137 | Active, not recruiting | Universidad de Zaragoza | 2020-12-30 |
| NCT04049760 | Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease | https://ClinicalTrials.gov/show/NCT04049760 | Recruiting | Amicus Therapeutics | 2022-06-01 |
| NCT04046224 | Dose-Ranging Study of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease | https://ClinicalTrials.gov/show/NCT04046224 | Recruiting | Sangamo Therapeutics | 2021-09-28 |
| NCT04040049 | A Fabry Disease Gene Therapy Study | https://ClinicalTrials.gov/show/NCT04040049 | Recruiting | Freeline Therapeutics | 2021-12-31 |
| NCT03737214 | A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease | https://ClinicalTrials.gov/show/NCT03737214 | Recruiting | Idorsia Pharmaceuticals Ltd. | 2022-05-22 |
| NCT03678324 | Pre-Clinical White Matter Changes and Associated Connectivity Effects in Fabry Disease | https://ClinicalTrials.gov/show/NCT03678324 | Recruiting | University of Utah | 2022-07-30 |
| NCT03614234 | Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients | https://ClinicalTrials.gov/show/NCT03614234 | Enrolling by invitation | Protalix | 2023-01-31 |
| NCT03566017 | Extension Study of 1 mg/kg Pegunigalsidase Alfa in Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT03566017 | Enrolling by invitation | Protalix | 2022-09-30 |
| NCT03500094 | Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years) | https://ClinicalTrials.gov/show/NCT03500094 | Active, not recruiting | Amicus Therapeutics | 2021-03-01 |
| NCT03454893 | Open-Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment -Naive Subjects With Classic Fabry Disease | https://ClinicalTrials.gov/show/NCT03454893 | Recruiting | AvroBio | 2021-07-31 |
| NCT03425539 | Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease | https://ClinicalTrials.gov/show/NCT03425539 | Recruiting | Idorsia Pharmaceuticals Ltd. | 2021-05-15 |
| NCT01218659 | Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT01218659 | Completed | Amicus Therapeutics | 2014-05-27 |
| NCT01196871 | Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease | https://ClinicalTrials.gov/show/NCT01196871 | Completed | Amicus Therapeutics | 2012-10-09 |
| NCT01178164 | Prevalence of Fabry’s Disease in a Population of Patients With Chronic Pains | https://ClinicalTrials.gov/show/NCT01178164 | Completed | University Hospital, Bordeaux | 2012-09-30 |
| NCT01124643 | Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT01124643 | Completed | Shire | 2013-07-08 |
| NCT00925301 | Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT00925301 | Completed | Amicus Therapeutics | 2012-06-12 |
| NCT00864851 | Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease | https://ClinicalTrials.gov/show/NCT00864851 | Completed | Shire | 2012-06-01 |
| NCT00230607 | Study of the Effects of Fabrazyme Treatment on Lactation and Infants | https://ClinicalTrials.gov/show/NCT00230607 | Recruiting | Sanofi | 2020-07-31 |
| NCT02798458 | Evaluation of the Gastrointestinal Manifestation of Fabry’s Disease | https://ClinicalTrials.gov/show/NCT02798458 | Recruiting | Massachusetts General Hospital | 2020-07-31 |
| NCT00701415 | A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms | https://ClinicalTrials.gov/show/NCT00701415 | Completed | Sanofi | 2015-06-30 |
| NCT00357786 | An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT00357786 | Completed | National Institutes of Health Clinical Center (CC) | 2008-12-31 |
| NCT00304512 | A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease | https://ClinicalTrials.gov/show/NCT00304512 | Completed | Amicus Therapeutics | 2008-05-09 |
| NCT00283959 | A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease | https://ClinicalTrials.gov/show/NCT00283959 | Completed | Amicus Therapeutics | 2008-05-08 |
| NCT00283933 | A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease | https://ClinicalTrials.gov/show/NCT00283933 | Completed | Amicus Therapeutics | 2008-03-12 |
| NCT00214500 | A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease | https://ClinicalTrials.gov/show/NCT00214500 | Completed | Amicus Therapeutics | 2008-01-29 |
| NCT00196716 | A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT00196716 | Completed | Sanofi | 2006-04-30 |
| NCT00140621 | A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease | https://ClinicalTrials.gov/show/NCT00140621 | Completed | Sanofi | 2012-08-31 |
| NCT00097890 | Replagal Enzyme Replacement Therapy for Adults With Fabry Disease | https://ClinicalTrials.gov/show/NCT00097890 | Completed | National Institutes of Health Clinical Center (CC) | NA |
| NCT00084084 | Replagal Enzyme Replacement Therapy for Children With Fabry Disease | https://ClinicalTrials.gov/show/NCT00084084 | Completed | Shire | 2011-06-30 |
| NCT00081497 | A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT00081497 | Completed | Sanofi | 2005-09-30 |
| NCT00075244 | Alternative Dosing and Regimen of Replagal to Treat Fabry Disease | https://ClinicalTrials.gov/show/NCT00075244 | Completed | National Institutes of Health Clinical Center (CC) | NA |
| NCT00074984 | A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease | https://ClinicalTrials.gov/show/NCT00074984 | Completed | Sanofi | 2004-01-31 |
| NCT00074971 | A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT00074971 | Completed | Sanofi | NA |
| NCT00074958 | A Study of Fabrazyme in Pediatric Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT00074958 | Completed | Sanofi | 2005-05-31 |
| NCT00071877 | An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease | https://ClinicalTrials.gov/show/NCT00071877 | Completed | National Institutes of Health Clinical Center (CC) | NA |
| NCT00068107 | Dosing Study of Replagal in Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT00068107 | Completed | Baylor Research Institute | 2013-12-31 |
| NCT00048906 | Alpha-Galactosidase A Replacement Therapy for Fabry Disease | https://ClinicalTrials.gov/show/NCT00048906 | Completed | National Institutes of Health Clinical Center (CC) | NA |
#If less then 500 studies
| nct_id | brief_title | link | overall_status | source | primary_completion_date |
|---|---|---|---|---|---|
| NCT03362164 | Evaluation of HEArt invoLvement in Patients With FABRY Disease | https://ClinicalTrials.gov/show/NCT03362164 | Recruiting | Wuerzburg University Hospital | 2032-03-31 |
| NCT03230149 | Prevalence and Characteristics of Fabry Disease (FD) in Patients With Stroke or Small Fiber Neuropathy | https://ClinicalTrials.gov/show/NCT03230149 | Recruiting | Assistance Publique - Hôpitaux de Paris | 2020-03-31 |
| NCT03135197 | German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl. | https://ClinicalTrials.gov/show/NCT03135197 | Active, not recruiting | University Hospital Muenster | 2020-07-31 |
| NCT02969200 | Fabry: Renal Function During Long-term ERT by 51Cr-EDTA Clearance | https://ClinicalTrials.gov/show/NCT02969200 | Completed | Rigshospitalet, Denmark | 2016-09-30 |
| NCT02908724 | Cardiac Involvement in Adult Patients With Fabry Disease; Relation to Enzyme Replacement Therapy | https://ClinicalTrials.gov/show/NCT02908724 | Completed | Rigshospitalet, Denmark | 2014-11-30 |
| NCT02778295 | Biomarker for Patients With Fabry Disease (BioFabry) | https://ClinicalTrials.gov/show/NCT02778295 | Active, not recruiting | Centogene AG Rostock | 2021-06-30 |
| NCT02719249 | Fabry Disease Screening in ESRD Patients in West of France | https://ClinicalTrials.gov/show/NCT02719249 | Completed | Rennes University Hospital | 2018-06-30 |
| NCT02469181 | Impact of Enzyme Replacement Therapy on Cardiac Function in Patients With Fabry’s Cardiomyopathy (RECAFTURE Trial) | https://ClinicalTrials.gov/show/NCT02469181 | Recruiting | Yonsei University | 2020-10-31 |
| NCT02152189 | Screening for Fabry Disease in a Pediatric Population at Risk | https://ClinicalTrials.gov/show/NCT02152189 | Completed | University Hospital Heidelberg | 2017-05-31 |
| NCT01295008 | Androgenetic Alopecia in Fabry Disease | https://ClinicalTrials.gov/show/NCT01295008 | Completed | Baylor Research Institute | 2015-10-31 |
| NCT01271699 | Prevalence of Fabry Disease in a Defined Population at Risk - Patients Formerly Diagnosed With Multiple Sclerosis | https://ClinicalTrials.gov/show/NCT01271699 | Completed | Centogene AG Rostock | 2019-12-01 |
| NCT01745185 | Immune Response in Subjects With Fabry Disease Who Are Switching From Agalsidase Alfa to Agalsidase Beta | https://ClinicalTrials.gov/show/NCT01745185 | Completed | O & O Alpan LLC | 2015-04-07 |
| NCT01632111 | Pulmonary Involvement in Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT01632111 | Completed | University of Zurich | 2014-05-31 |
| NCT01581424 | Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study | https://ClinicalTrials.gov/show/NCT01581424 | Recruiting | University of Minnesota | 2020-07-31 |
| NCT01355146 | Home Therapy With Replagal in Fabry Disease | https://ClinicalTrials.gov/show/NCT01355146 | Completed | Shire | 2017-09-30 |
| NCT04252066 | A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding | https://ClinicalTrials.gov/show/NCT04252066 | Recruiting | Amicus Therapeutics | 2029-08-31 |
| NCT04043273 | Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences | https://ClinicalTrials.gov/show/NCT04043273 | Recruiting | Amicus Therapeutics France SAS | 2022-09-30 |
| NCT03949920 | A Study of Migalastat in Fabry Disease | https://ClinicalTrials.gov/show/NCT03949920 | Recruiting | Manchester University NHS Foundation Trust | 2021-12-01 |
| NCT03886714 | Screening for Fabry Disease in Renal Transplantation | https://ClinicalTrials.gov/show/NCT03886714 | Recruiting | University Hospital, Montpellier | 2021-03-25 |
| NCT03838237 | Effect of Migalastat on Cardiac Involvement in Fabry Disease | https://ClinicalTrials.gov/show/NCT03838237 | Active, not recruiting | Ospedale San Donato | 2020-12-31 |
| NCT03683966 | MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study | https://ClinicalTrials.gov/show/NCT03683966 | Recruiting | Wuerzburg University Hospital | 2019-12-31 |
| NCT03596398 | Epidemiological Study of Fabry Disease in Taiwan Young Stroke Patients | https://ClinicalTrials.gov/show/NCT03596398 | Enrolling by invitation | Chiayi Christian Hospital | 2021-09-30 |
| NCT01268241 | The Efficacy and Safety of Switch Between Agalsidase Beta to Agalsidase Alfa for Enzyme Replacement in Patients With Anderson-Fabry Disease | https://ClinicalTrials.gov/show/NCT01268241 | Completed | Centogene AG Rostock | 2016-04-30 |
| NCT01210196 | Sophisticated Assessment of Disease Burden in Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT01210196 | Completed | Shire | 2013-09-30 |
| NCT01198899 | Belgian Screening Project for the Detection of Anderson-Fabry Disease in Hypertrophic Cardiomyopathy | https://ClinicalTrials.gov/show/NCT01198899 | Completed | University Hospital, Ghent | 2011-08-31 |
| NCT01182818 | Fabry and Stroke Epidemiological Protocol (FASEP): Risk Factors In Ischemic Stroke Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT01182818 | Completed | University of Rostock | 2012-04-30 |
| NCT01165697 | Establishment of Biomarkers for Fabry Disease | https://ClinicalTrials.gov/show/NCT01165697 | Completed | Ohio State University | 2014-07-31 |
| NCT01019629 | Fabry Screening Study | https://ClinicalTrials.gov/show/NCT01019629 | Completed | Baylor Research Institute | 2013-04-17 |
| NCT03230591 | Impact of Agalsidase Alfa Therapy on Cardiac funcTION in Patients With Fabry’s Cardiomyopathy | https://ClinicalTrials.gov/show/NCT03230591 | Recruiting | Yonsei University | 2019-06-30 |
| NCT04184986 | Screening of Fabry Disease in Patients With GI Symptoms | https://ClinicalTrials.gov/show/NCT04184986 | Recruiting | General University Hospital, Prague | 2020-09-01 |
| NCT00728364 | Anderson-Fabry Disease in Chronic Kidney Disease Patients Not on Renal Replacement Therapy | https://ClinicalTrials.gov/show/NCT00728364 | Completed | Klinikum Wels-Grieskirchen | 2011-08-31 |
| NCT00446862 | The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study | https://ClinicalTrials.gov/show/NCT00446862 | Completed | University of Alabama at Birmingham | 2012-06-30 |
| NCT00414583 | Stroke in Young Fabry Patients (sifap1): Frequency of Fabry Disease in Young Stroke Patients | https://ClinicalTrials.gov/show/NCT00414583 | Completed | Centogene AG Rostock | 2010-01-31 |
| NCT00413595 | Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation | https://ClinicalTrials.gov/show/NCT00413595 | Completed | Centogene AG Rostock | 2019-12-01 |
| NCT00343577 | Antiproteinuric Agents and Fabry Disease | https://ClinicalTrials.gov/show/NCT00343577 | Completed | University of Alabama at Birmingham | NA |
| NCT00233870 | A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease. | https://ClinicalTrials.gov/show/NCT00233870 | Completed | Sanofi | 2011-03-31 |
| NCT00168974 | Neuropathic Pain and Fabry Disease | https://ClinicalTrials.gov/show/NCT00168974 | Completed | Danish Pain Research Center | NA |
| NCT00106912 | Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity | https://ClinicalTrials.gov/show/NCT00106912 | Completed | National Institutes of Health Clinical Center (CC) | NA |
| NCT00005111 | PET Scans in Normal Volunteers and Patients With Fabry Disease | https://ClinicalTrials.gov/show/NCT00005111 | Completed | National Institutes of Health Clinical Center (CC) | NA |
| NCT00001774 | Vasodilation in Patients With Fabry’s Disease | https://ClinicalTrials.gov/show/NCT00001774 | Completed | National Institutes of Health Clinical Center (CC) | NA |
| NCT00030134 | Data Collection in Women With Fabry Disease | https://ClinicalTrials.gov/show/NCT00030134 | Completed | National Institutes of Health Clinical Center (CC) | NA |
| NCT00055016 | Registry of Fabry Disease - A Multicenter Observational Study | https://ClinicalTrials.gov/show/NCT00055016 | Completed | National Institutes of Health Clinical Center (CC) | NA |
| NCT00001491 | Analysis of the Nervous System in Patients With Fabry’s Disease | https://ClinicalTrials.gov/show/NCT00001491 | Completed | National Institutes of Health Clinical Center (CC) | NA |
#If less then 500 registries
| nct_id | brief_title | link | overall_status | source | primary_completion_date |
|---|---|---|---|---|---|
| NCT03321604 | Kidney Information Network for Disease Research and Education | https://ClinicalTrials.gov/show/NCT03321604 | Recruiting | Massachusetts General Hospital | 2022-01-31 |
| NCT03289065 | Fabry Outcome Survey (FOS) | https://ClinicalTrials.gov/show/NCT03289065 | Recruiting | Shire | 2030-04-01 |
| NCT02023086 | Clinical Investigation on the Blood Oxygenation at the Optic Nerve Head in Fabry Patients | https://ClinicalTrials.gov/show/NCT02023086 | Completed | Université de Montréal | 2016-09-30 |
| NCT00455104 | Canadian Fabry Disease Initiative (CFDI) National Registry | https://ClinicalTrials.gov/show/NCT00455104 | Recruiting | Canadian Fabry Research Consortium | 2029-10-31 |
| NCT00196742 | Fabry Disease Registry & Pregnancy Sub-registry | https://ClinicalTrials.gov/show/NCT00196742 | Recruiting | Sanofi | 2034-01-31 |